Heterogeneity and Gaps in Reporting Primary Outcomes From Neonatal Trials

Baba A 1 , Webbe J 2 , Butcher NJ 1 , Rodrigues C 1 , Stallwood E 1 , Goren K 1 Show all authors , Monsour A 1 , Chang ASM 3 , Trivedi A 4 , Manley BJ 5 , McCall E 6 , Bogossian F 7 , Namba F 8 , Schmölzer GM 9 , Harding J 10 , Nguyen KA 11 , Doyle LW 12 , Jardine L 13 , Rysavy MA 14 , Konstantinidis M 15 , Meyer M 16 , Helmi MAM 17 , Lai NM 18 , Hay S 19 , Onland W 20 , Choo YM 21 , Gale C 2 , Soll RF 22 , Offringa M 1 , Core Outcome Reporting in Neonatal Trials Study Group

Affiliations 

  • 1 Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, Ontario, Canada
  • 2 Neonatal Medicine, School of Public Health, Imperial College London, London, United Kingdom
  • 3 Quality, Safety and Risk Management, and Department of Neonatology, KK Women's and Children's Hospital, Singapore
  • 4 The Children's Hospital at Westmead, New South Wales, Australia
  • 5 The Royal Women's Hospital, Melbourne, Australia
  • 6 School of Nursing and Midwifery, Queen's University of Belfast, Belfast, Northern Ireland
  • 7 University of the Sunshine Coast, Sunshine Coast, Australia
  • 8 Department of Pediatrics, Saitama Medical Center, Saitama Medical University, Saitama, Japan
  • 9 Department of Pediatrics, University of Alberta, Edmonton, Alberta, Canada
  • 10 Liggins Institute, University of Auckland, Auckland, New Zealand
  • 11 Claude Bernard University Lyon, Villeurbanne, France
  • 12 Department of Obstetrics and Gynaecology, The Royal Women's Hospital, University of Melbourne, Parkville, Victoria, Australia
  • 13 Department of Neonatology, Mater Mothers' Hospital, South Brisbane, Queensland, Australia
  • 14 University of Texas Health Science Centre at Houston, Houston, Texas
  • 15 Institute of Health Policy, Management, and Evaluation, University of Toronto, Toronto, Ontario, Canada
  • 16 Middlemore Hospital, Auckland, New Zealand
  • 17 Department of Paediatrics, International Islamic University, Malaysia
  • 18 School of Medicine, Taylor's University, Malaysia
  • 19 Department of Neonatology, Beth Israel Deaconess Medical Center, Boston, Massachusetts
  • 20 Department of Neonatology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands
  • 21 Department of Paediatrics, University Malaya, Malaysia
  • 22 Cochrane Neonatal, Burlington, VT
Pediatrics, 2023 Sep 01;152(3).
PMID: 37641881 DOI: 10.1542/peds.2022-060751

Abstract

OBJECTIVES: Clear outcome reporting in clinical trials facilitates accurate interpretation and application of findings and improves evidence-informed decision-making. Standardized core outcomes for reporting neonatal trials have been developed, but little is known about how primary outcomes are reported in neonatal trials. Our aim was to identify strengths and weaknesses of primary outcome reporting in recent neonatal trials.

METHODS: Neonatal trials including ≥100 participants/arm published between 2015 and 2020 with at least 1 primary outcome from a neonatal core outcome set were eligible. Raters recruited from Cochrane Neonatal were trained to evaluate the trials' primary outcome reporting completeness using relevant items from Consolidated Standards of Reporting Trials 2010 and Consolidated Standards of Reporting Trials-Outcomes 2022 pertaining to the reporting of the definition, selection, measurement, analysis, and interpretation of primary trial outcomes. All trial reports were assessed by 3 raters. Assessments and discrepancies between raters were analyzed.

RESULTS: Outcome-reporting evaluations were completed for 36 included neonatal trials by 39 raters. Levels of outcome reporting completeness were highly variable. All trials fully reported the primary outcome measurement domain, statistical methods used to compare treatment groups, and participant flow. Yet, only 28% of trials fully reported on minimal important difference, 24% on outcome data missingness, 66% on blinding of the outcome assessor, and 42% on handling of outcome multiplicity.

CONCLUSIONS: Primary outcome reporting in neonatal trials often lacks key information needed for interpretability of results, knowledge synthesis, and evidence-informed decision-making in neonatology. Use of existing outcome-reporting guidelines by trialists, journals, and peer reviewers will enhance transparent reporting of neonatal trials.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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